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Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Design, Rationale, and Baseline Characteristics

Milind Y Desai1, Steve E Nissen2, Theodore Abraham3

  • 1Hypertrophic Cardiomyopathy Center, Cleveland, Ohio, USA; Cleveland Clinic Coordinating Center for Clinical Research, Heart, Vascular and Thoracic Institute, Cleveland Clinic, Cleveland, Ohio, USA.

JACC. Heart Failure
|February 5, 2025
PubMed

Insights

Mavacamten is being studied in the phase 3 ODYSSEY-HCM trial for symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). This trial evaluates mavacamten

Area of Science:

  • Cardiology
  • Clinical Trials
  • Pharmacology

Background:

  • Symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM) currently lacks approved therapeutic options.
  • Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease characterized by thickening of the heart muscle.
  • Nonobstructive HCM presents unique challenges in management and treatment development.

Purpose of the Study:

  • To evaluate the efficacy and safety of mavacamten in patients with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM).
  • To assess the impact of mavacamten on patient-reported health status and exercise capacity.
  • To provide baseline characteristics of patients enrolled in the ODYSSEY-HCM trial.

Main Methods:

  • The ODYSSEY-HCM trial is a phase 3, randomized, double-blind, placebo-controlled study.
  • The trial involves 580 randomized patients across 201 international sites.
  • Primary endpoints include changes in Kansas City Cardiomyopathy Questionnaire (KCCQ) score and peak oxygen consumption (pVO2) at 48 weeks.

Main Results:

  • Baseline characteristics of 580 randomized patients are described, with a mean age of 56 years.
  • Patients were symptomatic (70% NYHA class II, 30% class III) with a mean KCCQ score of 58.
  • The study population had a mean left ventricular ejection fraction of 66% and mean pVO2 of 18 mL/kg/min.

Conclusions:

  • The ODYSSEY-HCM trial is investigating mavacamten as a potential treatment for symptomatic nHCM.
  • Results will determine if mavacamten improves health status and exercise capacity in this patient population.
  • The study aims to establish a new therapeutic avenue for patients with nHCM.

Keywords:
characteristicsdesignmavacamtennonobstructive HCM

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